Gene Therapy, DNA’s Past, RNA’s Future: A Time of Promise

Graphical representation of DNA and genes 

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This story is part of a series on the latest developments in Regenerative Medicine. In 1999, I defined regenerative medicine as a collection of interventions that restore cells and organs that have been damaged by disease, injured by trauma, or that are time-consuming to function properly. I include many chemical, genetic, and protein drugs, cell-based therapies, and biological therapies that achieve that goal.

In these subgroups, we focus more on genetic therapies. We review the latest treatment options and review developments that aim to revolutionize healthcare. Each article in this collection focuses on a different aspect of gene therapy’s role within the larger narrative of Regenerative Medicine. 

The history of gene therapy is full of wisdom, imagination and research. The 1960s through the 1980s, in particular, was a time of great progress, marked by major breakthroughs and discoveries. During this time, there was a better understanding of the nature of genes and their ability to treat disease.

Today, gene therapy is at the forefront of genetic research, and the possibilities are endless. With continued research and development, gene therapy can change many lives.

Development of Gene Therapy Concepts

In the 1960s, the possibility of treating genetic disorders by generating therapeutic DNA sequences was explored. This theory gained momentum after the discovery in 1961. This research showed that messenger RNA, known as mRNA, is very important in transferring genetic information from DNA to protein factories by between cells.

This discovery came about as a result of a comprehensive study of bacteriophage T4, a virus that infects bacteria. The viral DNA was found to be transcribed into mRNA, the template for the synthesis of new viral particles. This method of writing and translation is now known as the central discipline of molecular biology.

Image obtained from the NCI CCR BIOINFORMATICS TRAINING AND EDUCATION PROGRAM Originally developed by the Children’s Research Hospital of St. Jude

Also, in 1961, Lorraine Kraus succeeded in inserting functional DNA into a mammalian cell. He genetically modified the hemoglobin of cells taken from the bone marrow of a patient with sickle-cell anemia. This is done by injecting the patient’s cells with DNA extracted from a donor with normal hemoglobin in cell culture. Shortly after this, these ideas were put into practical use.

Using Novel Gene Therapy Ideas

Gene Therapy, DNA's Past, RNA's Future: A Time of Promise

In 1972, two young sisters from West Germany were among the first to receive a pioneering gene therapy for a rare genetic disorder called hyperargininemia. This inherited condition is caused by a deficiency of the enzyme arginase, which leads to the accumulation of arginine in the blood. Any accumulation of arginine can cause brain damage, epilepsy and other nerve and muscle problems. Treatment was given as a last resort to save the children’s lives.

Gene therapy attempted to address the missing enzyme in the sisters by producing a modified enzyme. Unfortunately, the treatment was unsuccessful, and the sisters did not respond to the treatment. However, this first form of gene therapy highlighted the possibility of genetic intervention to treat inherited diseases. It paved the way for further research and development of ideas proposed years ago, although not everyone was on board with this pioneering approach.

Around this time, an influential paper was published about the potential of gene therapy to treat genetic diseases in humans. The authors suggested that DNA sequences could be inserted into patients’ cells for effective treatment. However, they warned that the scientific understanding of gene therapy was incomplete and needed to be addressed, making it a difficult and dangerous treatment option.

Genetic Engineering and Retroviruses

Hot on the heels of these efforts, the field of genetic engineering flourished and provided new tools that can be used in gene therapy. One of these new tools was retroviruses. These viruses were a more efficient way to transfer genes.entering the nucleus of the cell in a vector way.

Image from Your Genome | The original was developed by Genome Research Limited

Richard Mulligan, a researcher at the Massachusetts Institute of Technology, developed the first viral vector suitable for gene therapy. In 1983, in collaboration with colleagues, Mulligan genetically modified the mouse leukemia retrovirus. A retrovirus is modified to provide the desired DNA without replicating in humans. The selected marker, a fragment of DNA from the bacterium Escherichia coli, is inserted into the new vector. This marker helps determine the number of genes a cell has received during gene transfer. Mulligan and his colleagues have published the functions of these retroviruses, how to use them to express selected genes in cells, and how to transfer genes efficiently. These discoveries allowed the field to rapidly advance with new experiments and cases.

Building Success

In the late 1980s, French Anderson conducted a groundbreaking trial to treat patients with ADA-SCID, a severe genetic disease that destroys the immune system. Anderson’s experiment involved inserting a functional copy of the defective gene into the patient’s cells using a virus as a carrier. This approach marked the first successful clinical demonstration that gene therapy could be used to treat genetic diseases. Anderson’s pioneering work opened new therapeutic possibilities for the treatment of genetic diseases and made him the “father of gene therapy.” 

This was followed by publications of the “first human genetics experiment”. This was an important step in genetic research. The experiment described involved using a retrovirus to deliver a working copy of a missing or defective gene into a patient’s cells. This method allowed the modified genes to be inserted into the patient’s cells, creating a new source of functional proteins that can combat the genetic problem. The experiment demonstrated the viability of retroviral genes in humans, paving the way for further advances in genetic research and new methods of treating genetic diseases.

The success of these early experiments led to an explosive development in genetic research.

The Promised Time Will Set the Frame

From 1960 to 1980, there has been a lot of research and development of gene therapy, the great success of gene therapy. In these two decades, amazing strides in scientific understanding have been made, such as elucidating mRNA and conducting the first experiments in humans. This period marked a time of real promise.

The next decade was not so kind to gene therapy. In the 1990s, the field faced many challenges and obstacles that threatened to hinder progress. Despite these challenges, the spirit of innovation and innovation that defined the early years of gene therapy has not wavered, and we continue to build on its rich legacy. .

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He is a scientist, businessman, author, and philanthropist. For nearly two decades, I was a professor at Harvard Medical School and the Harvard School of Public Health where I established two academic research departments, the Division of Biochemical Pharmacology and the Division of Human Retrovirology. I am probably best known for my work on cancer, HIV/AIDS, genomics, and today, on COVID-19. My biography, My Lifelong Fight Against Disease, published in October. I am the chairman and president of ACCESS Health International, a non-profit organization I founded that promotes innovative solutions to the health challenges of our time. Each of my articles on will focus on a specific health care challenge and provide best practices and innovative solutions to overcome those challenges for the benefit of all.

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